A Double-Blind, Randomized, Placebo-Controlled Phase II Clinical Trial to Evaluate the Safety and Efficacy of L2-01 in Subjects With Crohn's Disease

NCT07010926 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: L2-01-CD
• Study Type: interventional
• Target: 120
• Locations: 0 countries
• Sites: N/A

Brief Summary

This clinical trial is testing a new treatment called L2-01 in patients with Crohn's Disease, a condition that causes ongoing inflammation of the digestive tract. Current treatments for Crohn's Disease do not work for everyone, and some patients still experience symptoms even with standard therapies. The purpose of this study is to determine if a single intravenous infusion of L2-01, which uses cells called mesenchymal stem cells derived from a patient's own or their close relative's body fat, is safe and can help improve symptoms of Crohn's Disease. Researchers will compare the effects of L2-01 to a placebo (an inactive substance) to see if L2-01 helps reduce inflammation and improves quality of life in patients. The main questions the study aims to answer are: Is L2-01 safe for patients with Crohn's Disease? Can L2-01 effectively reduce symptoms and improve the health of people living with Crohn's Disease compared to a placebo? The researchers believe that L2-01 will be safe and more effective than placebo in reducing inflammation, decreasing disease activity, and improving patients' quality of life.

Conditions

Crohn&Amp;#39;s; Crohn&Amp;#39;s Disease (CD)

Keywords

Crohn's Disease; Stem Cells; Autologous; Mesenchymal Stem Cells (MSC); Adipose; Crohn's

Outcome Measures

Primary Outcomes (1)

• Incidence of adverse events occurring in less than 5% of study participants as a measure of safety.

  From the day of infusion through 12 weeks post-infusion.

Secondary Outcomes (5)

• Change in Crohn's Disease Activity Index (CDAI) from baseline to 12 weeks post-infusion.

  Baseline and 12 weeks post-infusion.

• Improvement in inflammatory markers and MRI results from baseline to 12 weeks post-infusion.

  Baseline and 12 weeks post-infusion.

• Change in quality of life as assessed by the Inflammatory Bowel Disease Questionnaire (IBDQ-32) from baseline to 12 weeks post-infusion.

  Baseline and 12 weeks post-infusion.

• Relapse rate among patients who achieved treatment success with adipose-derived mesenchymal stem cells.

  Up to 24 weeks post-infusion.

• Biological characterization of the cell product (phenotype study, suppressor capacity, cytokine production) and its correlation with therapeutic effect.

  At infusion and during follow-up assessments up to 24 weeks.

Other Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs), withdrawals due to adverse events, changes in laboratory values, and vital signs.

  From day of infusion through 24 weeks post-infusion.

Study Arms (2)

L2-01 Treatment Arm

EXPERIMENTAL

Participants in this arm receive a single intravenous infusion of L2-01, a human adipose-derived mesenchymal stem cell product. The dose is 1 × 10\^6 MSC cells per kilogram of body weight, suspended in 10 ml of lactated Ringer's solution mixed with 100 ml of saline. The infusion is administered over approximately 30 minutes. MSCs are derived from the patient's own adipose tissue or from that of a first-degree relative.

Biological: L2-01 adipose-derived mesenchymal stem cells (MSC)

Placebo Comparator Arm

PLACEBO COMPARATOR

Participants in this arm receive a single intravenous infusion of placebo, consisting of 10 ml of lactated Ringer's solution mixed with 100 ml of saline. The infusion is administered over approximately 30 minutes. The placebo product is packaged identically to the active product to maintain study blinding.

Other: Placebo saline solution

Interventions

L2-01 adipose-derived mesenchymal stem cells (MSC) BIOLOGICAL

L2-01 is a biological product consisting of human mesenchymal stem cells isolated from adipose tissue of the patient or a first-degree relative. The MSCs are expanded under Good Manufacturing Practice (GMP) conditions and suspended in 10 ml of lactated Ringer's solution. The product is administered as a single intravenous infusion at a dose of 1 × 10\^6 cells per kilogram of body weight, diluted in 100 ml of saline, infused over approximately 30 minutes. The infusion is closely monitored for safety.

L2-01 Treatment Arm

Placebo saline solution OTHER

Placebo consists of 10 ml of lactated Ringer's solution mixed with 100 ml of saline. It is administered as a single intravenous infusion over approximately 30 minutes. The placebo is packaged identically to the active product to maintain study blinding.

Placebo Comparator Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with Crohn Disease (CD) diagnosed within 12 months before acceptance of clinical, endoscopical, anatomopathological and/or radiological criteria and have a non-active CD. (Crohn´s Disease Activity Index (CDAI)≤ 200)
• Any gender, \> 18 Years of age
• Negative pregnancy test in female subjects
• Patients voluntarily sign the informed consent before performance of any study-related procedures
• Patient is, in the investigator's opinion, willing and able to comply with the protocol requirements

You may not qualify if:

• Patients who have received infliximab or any other anti-TNF agent within 8 weeks before the cell treatment administration.
• Patients who have received tacrolimus or cyclosporine within 4 weeks before cell treatment.
• Severe uncontrolled diseases (chronic renal failure, cardio, pulmonary, etc.).
• Patients with a diagnosis of malignant neoplasia, except basal cell or epidermoid carcinoma of the skin, or a previous history of malignant tumors, except those that have no evidence of relapse for at least 5 years.
• Subjects with congenital or acquired immunodeficiency.
• Subjects who tested positive for HIV 1/2, HBV, HCV, HTLV I/II, WNV, and syphilis
• Flu symptoms
• Patient had major surgery or serious trauma within 6 weeks before enrolment.
• Pregnant or breastfeeding women.
• Physical or psychological impossibility of following the protocol requirements
• Patients who are receiving or have received another investigational drug within 30 days prior to the first visit.

Sponsors & Collaborators

• L2 Bio, LLC: lead
• FDAMap: collaborator
• Akan Biosciences, Inc.: collaborator

Study Officials

• Mukesh Kumar, PhD, RAC

  FDAMap

  STUDY DIRECTOR

Central Study Contacts

Vince Liguori

CONTACT

847-561-0217; vince@agebiomed.com

Mukesh Kumar, PhD, RAC

CONTACT

240-750-4893; mkumar@fdamap.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Study monitors will also be blinded
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blind, placebo controlled

Study Record Dates

• First Submitted: May 29, 2025
• First Posted: June 8, 2025
• Study Start: August 15, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: June 8, 2025

Record last verified: 2025-05